One of the world's top ex­perts in coro­nary heart dis­ease is spear­head­ing a new gene edit­ing up­start out to trans­form the field

As head of the Cen­ter for Hu­man Ge­net­ic Re­search at Mass­a­chu­setts Gen­er­al Hos­pi­tal and the Broad’s Car­dio­vas­cu­lar Dis­ease Ini­tia­tive, Sekar Kathire­san has oc­cu­pied a sin­gu­lar po­si­tion as one of the world’s lead­ing ex­perts on the con­nec­tion be­tween ge­net­ics and coro­nary heart dis­ease. He’s tracked how peo­ple dealt a bad ge­net­ic hand — and the el­e­vat­ed risks that come with it — can lim­it in­her­ent dan­gers by lifestyle changes, and pon­dered over the ef­fects of dai­ly drugs used to treat mass pa­tient groups. And he’s reached a sim­ple con­clu­sion:

None of it is re­al­ly work­ing. 

In par­tic­u­lar, none of that is any­where near as use­ful as the ge­net­ic mu­ta­tions that he’s seen that con­fer a low­er risk of dy­ing and car­dio events. Par­tic­u­lar­ly the in­di­vid­u­als who car­ry mu­ta­tions “which break ei­ther of two genes — APOC3 or ANGPTL3 — rapid­ly clear triglyc­eride-rich lipopro­teins from the cir­cu­la­tion” and pro­tect them from heart at­tack.

Now, he’s mak­ing a pro­fes­sion­al leap to see if he and a squad of in­ves­ti­ga­tors at a new­ly launched biotech can dra­mat­i­cal­ly change the im­per­fect sta­tus quo through gene edit­ing.

“Imag­ine,” he tells me, “an in­jec­tion ad­min­is­tered once in life that safe­ly con­fers last­ing pro­tec­tion.”

No more pills. No fleet­ing com­mit­ments to healthy lifestyles that can’t stretch past Jan­u­ary. But a wide­spread and durable shar­ing of the same health ben­e­fits he’s seen in the very, very few. That’s the dream.

To­day, Kathire­san is step­ping down from his lofty aca­d­e­m­ic roles and mak­ing his de­but as CEO of Verve Ther­a­peu­tics, which is tak­ing its place in the hotbed of gene ther­a­py work around Cam­bridge, MA. The small team of 10 — soon to dou­ble in size — may not come close to ri­val­ing the big biotechs that oc­cu­py the streets in and around Har­vard and MIT. But rel­a­tive­ly few can claim the same kinds of con­nec­tions in the realms of drug sci­ence.

Ki­ran Musunuru

An­oth­er car­dio ge­net­ics ex­pert, Penn’s Ki­ran Musunuru and Har­vard pro­fes­sor J Kei­th Joung, who co-found­ed gene ther­a­py pi­o­neer Ed­i­tas, are on board as sci­en­tif­ic co-founders. There’s an al­liance with Beam Ther­a­peu­tics, the up­start next-gen gene edit­ing out­fit found­ed by Feng Zhang, one of 3 sci­en­tists wide­ly cred­it­ed with ush­er­ing in the CRISPR rev­o­lu­tion that has armed re­searchers around the world with ef­fec­tive tools to ac­com­plish their work. And Ver­i­ly will help work on the nanopar­ti­cles they plan to use for de­liv­ery.

Burt Adel­man, the for­mer EVP of R&D at Bio­gen, will chair the board, which in­cludes the Broad’s chief da­ta of­fi­cer, An­tho­ny Philip­pakis.

Beam will pro­vide some of the tech, and has an op­tion to step in on fu­ture com­mer­cial­iza­tion. Verve has al­so nailed down CRISPR patents, in­clud­ing Cas9 and Cas12a (Cpf1), from the Broad In­sti­tute and Har­vard Uni­ver­si­ty.

And they have $58.5 mil­lion in cash to do their work from GV (you prob­a­bly still think of them as Google Ven­tures), which is step­ping in with ARCH Ven­ture Part­ners, F-Prime Cap­i­tal, and Bio­mat­ics Cap­i­tal to form the orig­i­nal syn­di­cate.

To be suc­cess­ful, the Verve team un­der Kathire­san will not just have to demon­strate their ap­proach can safe­ly work, but al­so that it ul­ti­mate­ly can be done on a mass ba­sis in eco­nom­ic terms. That’s a tall or­der, but they do have some ad­van­tages, per­haps most no­tice­ably the ad­vances the ground­break­ers have made at the FDA, says the sci­en­tist.

“Gene edit­ing has the po­ten­tial to com­plete­ly trans­form the treat­ment par­a­digm for the dis­ease,” says Musunuru. “Pre­clin­i­cal stud­ies con­duct­ed in the field, in­clud­ing work done in my lab, have shown the promise of gene edit­ing to safe­ly re­duce cho­les­terol and oth­er coro­nary artery dis­ease risk fac­tors.”

So far, gene edit­ing in the le­git­i­mate R&D world has been cen­tered on the painstak­ing ad­vances of a hand­ful of pro­grams aimed at rare dis­eases. And Verve will start with its own rare ail­ments, tar­get­ing pa­tient pop­u­la­tions with the high­est un­met med­ical need. But Kathire­san isn’t drop­ping his pres­ti­gious aca­d­e­m­ic roles to search for mar­gin­al gains. He wants to tack­le the whole threat on a world­wide ba­sis.

That, of­fi­cial­ly, starts at Verve to­day.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll